Department of Biomedical Sciences
Helen Goodridge, PhD
Seigo Hatada, PhD
All blood cells are derived from blood stem cells. Red blood cells transport oxygen; white blood cells fight infections, kill tumor cells, and repair damaged tissues; and platelets cause blood to clot to stop bleeding. The Blood Program at the Regenerative Medicine Institute (RMI) is using stem cell technology to translate basic science research into safer therapeutic strategies for a broad range of blood-based illnesses and pioneering gene-targeting methods to correct disease-causing mutations.
Anemia afflicts more than 3 million people in the United States; some types (e.g., sickle cell anemia, aplastic anemia, and thalassemia) are severe and difficult to treat. More than 150 primary immunodeficiency disorders affecting white blood cell function render patients susceptible to life-threatening infections. Nearly 1 million people in the United States are living with or are in remission from blood cell cancers (e.g., leukemia, lymphoma, and myeloma).
Mutations in blood cells cause anemias, immunodeficiencies, and blood cancers Defective white blood cell function increases susceptibility to infections and tumors; overactive white blood cells cause allergies and inflammatory diseases. Blood stem cell transplants are already used to treat blood diseases and cancer, but immune rejection of donor cells and immune attack by donor white blood cells (graft-versus-host disease) remain serious risks.
By defining the molecular mechanisms that control white blood cell production from stem cells, we are identifying therapeutic targets to boost immune function. We are pioneering new genetic engineering strategies to remove genes and correct mutations that cause cancer (e.g., leukemia) and blood diseases (e.g., thalassemia). In addition to blood stem cells, we are using pluripotent stem cells produced by the RMI Induced Pluripotent Stem Cell (iPSC) Core Facility. Developing strategies that use a patient’s own cells will minimize the risks associated with blood stem cell transplants. We are partnering with Cedars-Sinai hematologists and oncologists to share knowledge and accelerate the pace of discovery.
ON THE HORIZON
Our research will pave the way for innovative therapeutic approaches to promote immune responses against infections and tumors, correct mutations that cause blood diseases, and prevent rejection of transplanted cells and tissues. We are committed to improving the safety and efficacy of stem cell transplants to provide long-term or even permanent solutions to a variety of blood diseases.